Trial Outcomes & Findings for A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) (NCT NCT02292537)
NCT ID: NCT02292537
Last Updated: 2021-02-17
Results Overview
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
126 participants
Primary outcome timeframe
Baseline and Month 15
Results posted on
2021-02-17
Participant Flow
After parental informed consent was obtained and prior to any treatment, participants entered a Screening Period of up to 21 days to determine their eligibility for the study. Of the 179 participants screened, 53 were screening failures.
Participant milestones
| Measure |
Sham Procedure
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Overall Study
STARTED
|
42
|
84
|
|
Overall Study
Completed Treatment
|
42
|
83
|
|
Overall Study
COMPLETED
|
42
|
84
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
Baseline characteristics by cohort
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
Total
n=126 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
3.4 years
STANDARD_DEVIATION 1.61 • n=5 Participants
|
3.8 years
STANDARD_DEVIATION 1.63 • n=7 Participants
|
3.6 years
STANDARD_DEVIATION 1.63 • n=5 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
67 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=5 Participants
|
38 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
0 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
42 Participants
n=5 Participants
|
80 Participants
n=7 Participants
|
122 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
7 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
30 Participants
n=5 Participants
|
64 Participants
n=7 Participants
|
94 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Multiple
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
|
19.9 scores on a scale
STANDARD_DEVIATION 7.23 • n=5 Participants
|
22.4 scores on a scale
STANDARD_DEVIATION 8.33 • n=7 Participants
|
21.6 scores on a scale
STANDARD_DEVIATION 8.03 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and Month 15Population: ITT Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Missing postbaseline HFMSE data were imputed using the multiple imputation method.
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15
|
-1.0 scores on a scale
Interval -2.5 to 0.5
|
3.9 scores on a scale
Interval 3.0 to 4.9
|
SECONDARY outcome
Timeframe: Baseline and Month 15Population: ITT Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Missing postbaseline HFMSE data were imputed using multiple imputation.
The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0=being unable, 1=performed with some compensation, and 2=unaided. The overall score is the sum of the scores for all activities with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15
|
26.3 Proportion of participants
Interval 12.4 to 40.22
|
56.8 Proportion of participants
Interval 45.62 to 68.05
|
SECONDARY outcome
Timeframe: Month 15Population: Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data.
New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone.
Outcome measures
| Measure |
Sham Procedure
n=34 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=66 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Proportion of Participants That Achieved Any New Motor Milestone at Month 15
|
5.9 Proportion of participants
Interval 0.72 to 19.68
|
19.7 Proportion of participants
Interval 10.93 to 31.32
|
SECONDARY outcome
Timeframe: Month 15Population: Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data.
New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone.
Outcome measures
| Measure |
Sham Procedure
n=34 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=66 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of New Motor Milestones Achieved Per Participant
|
-0.2 milestones achieved
Standard Deviation 0.54
|
0.2 milestones achieved
Standard Deviation 0.51
|
SECONDARY outcome
Timeframe: Baseline and Month 15Population: ITT Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Missing postbaseline data were imputed using multiple imputation.
The RULM Test is used in patients with SMA to assess upper limb functional ability items that are reflective of activities of daily living (i.e., raise a can to mouth as if drinking, take a coin and place it in a box, remove the lid of a container). The RULM test has a total of 20 items with an entry item that serves as functional class identification and does not contribute to the total score. The remaining 19 scorable items reflect different functional domains and are graded on a 3-point system with a score of 0 (unable), 1 (able, with modification), and a maximum of 2 (able, no difficulty). There is only 1 item (item I) that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for a total score range of 0-37, with higher scores increased great upper limb function. A positive change from Baseline indicates improvement.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Change From Baseline in Revised Upper Limb Module (RULM) Test
|
0.5 scores on a scale
Interval -0.6 to 1.6
|
4.2 scores on a scale
Interval 3.4 to 5.0
|
SECONDARY outcome
Timeframe: Month 15Population: Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data.
If the participant was unable to achieve standing alone at Baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder.
Outcome measures
| Measure |
Sham Procedure
n=34 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=66 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Proportion of Participants That Achieved Standing Alone
|
2.9 Proportion of participants
Interval 0.07 to 15.33
|
1.5 Proportion of participants
Interval 0.04 to 8.16
|
SECONDARY outcome
Timeframe: Month 15Population: Efficacy Set: All participants with a Day 456 Visit and all participants with a time difference of at least 463 days (456 days plus a 7-day window) between the date of first dose and the date for the final analysis. Based on imputed data where there was missing data.
If the participant was unable to achieve walking with assistance at baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder.
Outcome measures
| Measure |
Sham Procedure
n=34 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=66 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Proportion of Participants That Achieved Walking With Assistance
|
0.0 Proportion of participants
Interval 0.0 to 10.28
|
1.5 Proportion of participants
Interval 0.04 to 8.16
|
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. All participants with AEs were reported in this outcome measure, whereas in Adverse Event section there was at 5% reporting threshold to be met.
AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death; an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity. Any other medically important event that, in the opinion of the investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs)
AEs
|
42 participants
|
78 participants
|
|
Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs)
SAEs
|
12 participants
|
14 participants
|
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Any new or worsening vital sign findings were reported as AEs and are presented in the AE/SAE section of the results.
Vital signs assessed for clinical significance include resting blood pressure, pulse, respiratory rate, and temperature.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Systolic blood pressure <90 mmHg
|
33 participants
|
80 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Systolic blood pressure >140 mmHg
|
1 participants
|
4 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Systolic blood pressure >160 mmHg
|
0 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Diastolic blood pressure <50 mmHg
|
31 participants
|
65 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Diastolic blood pressure >90 mmHg
|
7 participants
|
11 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Diastolic blood pressure >100 mmHg
|
2 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Heart rate <60 beats/min
|
0 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Heart rate >100 beats/min
|
42 participants
|
84 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Temperature >38.0 C
|
4 participants
|
10 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Temperature <36.0 C
|
14 participants
|
33 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Respiratory rate <12 breaths/min
|
0 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Vital Sign Abnormalities
Respiratory rate >20 breaths/min
|
42 participants
|
84 participants
|
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Any new or worsening weight abnormality findings were reported as AEs and are presented in the AE/SAE section of the results.
Weight changes assessed from Baseline to Month 15.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of Participants With Clinically Significant Weight Abnormalities
Weight decrease of >=7% from baseline
|
3 participants
|
4 participants
|
|
Number of Participants With Clinically Significant Weight Abnormalities
Weight increase of >=7% from baseline
|
38 participants
|
77 participants
|
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Neurological examination clinical significance was not collected.
Neurological changes assessed for clinical significance include assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Physical examination clinical significance was not collected.
Physical examination changes were assessed for clinical significance.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure. Any new or worsening clinical laboratory parameter findings were reported as AEs and are presented in the AE/SAE section of the results.
Laboratory parameter changes assessed for clinical significance include serum chemistry, hematology, coagulation and urinalysis.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Hemoglobin - High
|
2 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Hemoglobin - Low
|
7 participants
|
15 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Hematocrit - High
|
3 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Hematocrit - Low
|
6 participants
|
18 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Bilirubin - High
|
1 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Total bilirubin - direct - Low
|
5 participants
|
8 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Total bilirubin - direct - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Total bilirubin - indirect- Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Total bilirubin - indirect- High
|
1 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Alkaline phosphatase - Low
|
1 participants
|
10 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Alkaline phosphatase - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: ALT - Low
|
0 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: ALT - High
|
4 participants
|
3 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: AST - Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: AST- High
|
2 participants
|
3 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: BUN - Low
|
0 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: BUN - High
|
1 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Total protein - Low
|
1 participants
|
7 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Total protein - High
|
9 participants
|
13 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Albumin - Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Albumin - High
|
19 participants
|
27 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Calcium - Low
|
0 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Calcium - High
|
9 participants
|
20 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Phosphorus - Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Phosphorus - High
|
8 participants
|
8 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Bicarbonate - Low
|
11 participants
|
29 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Bicarbonate - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Glucose - Low
|
7 participants
|
17 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Glucose - High
|
26 participants
|
44 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Cystatin C - Low
|
18 participants
|
36 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Cystatin C -High
|
0 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: CPK - Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: CPK - High
|
10 participants
|
16 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Specific gravity - Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Specific gravity - High
|
2 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: pH- Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: pH - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Protein - High
|
14 participants
|
43 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Glucose - High
|
2 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Ketones - High
|
18 participants
|
29 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Bilirubin - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Blood - High
|
1 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: RBC - High
|
1 participants
|
6 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: WBC - High
|
4 participants
|
13 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Epithelial Cells - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Bacteria - High
|
15 participants
|
24 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Casts - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Hyaline casts - High
|
1 participants
|
5 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Crystals - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Amorphous crystals - High
|
10 participants
|
16 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Red blood cells - High
|
2 participants
|
2 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Red blood cells - Low
|
7 participants
|
13 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: White blood cells - High
|
11 participants
|
12 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: White blood cells - Low
|
10 participants
|
20 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Platelet Count - High
|
5 participants
|
10 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Hematology: Platelet count - Low
|
10 participants
|
15 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Bilirubin - Low
|
19 participants
|
36 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Creatinine - Low
|
17 participants
|
39 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Creatinine- High
|
0 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Sodium - Low
|
1 participants
|
1 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Sodium - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Potassium - Low
|
2 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Potassium - High
|
1 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Chloride - Low
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Blood Chemistry: Chloride - High
|
0 participants
|
0 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Calcium oxalate crystals - High
|
17 participants
|
26 participants
|
|
Number of Participants With Clinically Significant Laboratory Parameter Abnormalities
Urinalysis: Uric acid crystals - High
|
5 participants
|
5 participants
|
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure.
The number of participants with abnormal, clinically relevant worsening, defined as participants with an ECG interpreted as abnormal and clinically relevant, with a comparison with Baseline value is reported.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of Participants With Abnormal, Clinically Relevant Post-Baseline Worsening in Electrocardiogram (ECG) in Results
|
2 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Baseline through Month 15Population: Safety Set: All participants who were randomized and received at least 1 dose of study drug/sham procedure.
Concomitant medications include prescription and over-the-counter medications administered to participants on or after the first day of study treatment.
Outcome measures
| Measure |
Sham Procedure
n=42 Participants
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 Participants
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Number of Participants Taking Any Concomitant Medication Related to Dosing Procedure or Sham Procedure
|
42 participants
|
84 participants
|
Adverse Events
Sham Procedure
Serious events: 12 serious events
Other events: 42 other events
Deaths: 0 deaths
Nusinersen
Serious events: 14 serious events
Other events: 75 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Sham Procedure
n=42 participants at risk
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 participants at risk
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Gastrointestinal disorders
Constipation
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Gastrointestinal disorders
Faecaloma
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
General disorders
Pain
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Bronchitis
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Gastroenteritis
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
2.4%
2/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Influenza
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Parainfluenzae virus infection
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Pneumonia
|
14.3%
6/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
2.4%
2/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Pneumonia adenoviral
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Pneumonia parainfluenzae viral
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Pneumonia viral
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
3.6%
3/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Respiratory syncytial virus bronchitis
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Upper respiratory tract infection
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Metabolism and nutrition disorders
Dehydration
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
1.2%
1/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
2.4%
2/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.4%
1/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
0.00%
0/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
Other adverse events
| Measure |
Sham Procedure
n=42 participants at risk
Sham comparator on Days 1, 29, 85 and 274.
|
Nusinersen
n=84 participants at risk
Nusinersen 12 mg solution via intrathecal (IT) injection on Days 1, 29, 85 and 274.
|
|---|---|---|
|
Gastrointestinal disorders
Constipation
|
9.5%
4/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
6.0%
5/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Gastrointestinal disorders
Diarrhoea
|
7.1%
3/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
9.5%
8/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Gastrointestinal disorders
Vomiting
|
11.9%
5/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
28.6%
24/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
General disorders
Pyrexia
|
35.7%
15/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
42.9%
36/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Bronchitis
|
9.5%
4/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
8.3%
7/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Conjunctivitis
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
7.1%
6/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Ear infection
|
11.9%
5/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
8.3%
7/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Gastroenteritis
|
19.0%
8/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
10.7%
9/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Gastroenteritis viral
|
9.5%
4/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
9.5%
8/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Influenza
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
9.5%
8/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Nasopharyngitis
|
35.7%
15/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
23.8%
20/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Otitis media
|
9.5%
4/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
6.0%
5/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Pharyngitis streptococcal
|
7.1%
3/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
4.8%
4/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Pneumonia
|
14.3%
6/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
4.8%
4/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Infections and infestations
Upper respiratory tract infection
|
45.2%
19/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
29.8%
25/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.5%
4/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
4.8%
4/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
25.0%
21/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Musculoskeletal and connective tissue disorders
Joint contracture
|
16.7%
7/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
4.8%
4/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.1%
3/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
4.8%
4/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
7.1%
3/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
3.6%
3/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Nervous system disorders
Headache
|
7.1%
3/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
28.6%
24/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
21.4%
9/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
25.0%
21/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
7.1%
6/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
16.7%
7/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
7.1%
6/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract congestion
|
4.8%
2/42 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
6.0%
5/84 • From signing of Informed Consent to the end of the Follow-up period (Month 15)
Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
- Publication restrictions are in place
Restriction type: OTHER